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Sickle Cell Disease Treatment Demonstration Program

Gerald Woods, MD, Division Director of Hematology/Oncology/BMT, has more than 30 years experience in sickle cell treatment. Dr. Woods is currently a PI for a study of GBT-440, a new drug to treat sickle cell.

Children’s Mercy is one of only two U.S. hospitals initially invited to participate in this research. Dr. Woods is leading the hospital’s participation in a Health Resources and Services Administration grant for the Sickle Cell Disease Treatment Demonstration Program Regional Collaborative.

Join us as Dr. Woods discusses the latest advancements in sickle cell disease and efforts to increase treatment options.

Sickle Cell Disease Treatment Demonstration Program
Featured Speaker:
Gerald M. Woods, MD
Gerald Woods, MD, is the Division Director of Hematology/Oncology and Bone Marrow Transplant at Children’s Mercy Kansas City and serves as Section Chief of Hematology, and director of the Sickle Cell Program. Dr. Woods also is a professor of Pediatrics at the University of Missouri-Kansas City School of Medicine. Dr. Woods received his medical degree from Duke University Medical Center. He completed his residency in Pediatrics at Children’s Mercy and returned to Duke for his fellowship in Pediatric Hematology/Oncology. Dr. Woods has been with Children’s Mercy now for more than 30 years.

Learn more about Gerald Woods, MD
Transcription:
Sickle Cell Disease Treatment Demonstration Program

Dr. Michael Smith (Host): Alright, so today we’re going to talk about Sickle Cell Disease Treatment Demonstration Program. My guest is Gerald Woods. He is the Division Director of Hematology and Oncology and Bone Marrow Transplant at Children’s Mercy Kansas City, and he’s the Director of the Sickle Cell Program. Dr. Woods, welcome to the show.

Dr. Gerald Woods (Guest): Thank you for having me, Dr. Smith.

Dr. Smith: Yeah, so let’s talk – I found in your bio, Dr. Woods, that you have been treating Sickle Cell Disease for 30 plus years and so I just have to – over the years, how have things evolved? And where are we at today in treating Sickle Cell?

Dr. Woods: I think we’re a lot farther along when I started getting involved with Sickle Cell Disease and that even goes back to my college days when I did my thesis on Sickle Cell Disease trying to learn more. We still only have one cure for Sickle Cell Disease, and that’s bone marrow transplant, which is not available for everyone, and it’s not an easy procedure to undergo, but over time, we’ve developed more refined supportive care, diagnostic services, where we can improve the quality of life and the longevity of patients with Sickle Cell Disease, so that has been encouraging. I think we may be on the cusp with some genetic modifications, hopefully coming up with another type of cure other than bone marrow transplant.

Dr. Smith: Yeah, that was going to be my next question for you, Dr. Woods, is where is a lot of that genetic research that we’re doing, genetic – gene therapy – is there hope that that’s going to be something that’s going to be able to maybe even cure Sickle Cell at some point?

Dr. Woods: I think so, but 20 years ago I said that too because we thought we were getting closer. We ran into some obstacles. It’s really – you have to have a vector where you can introduce a “normal” gene to replace the abnormal gene, and that was harder than I just thought it would be, but now we’re making more progress with other blood disorders like Hemophilia, Alpha Thalassemia, and Sickle Cell. The American Society of Hematology Annual meeting this past December, there were several reports where it’s much more encouraging that we’re getting so much closer. I think that will probably, hopefully, happen in my lifetime and we’ll improve the overall care for Sickle Cell patients. Once again, it’s probably not going to be for everyone, but at least it will be a good start point.

Dr. Smith: Now, you’re the primary investigator for a new drug, GBT440, tell us about that.

Dr. Woods: The only FDA-approved modifying therapy for Sickle Cell – so it’s not a cure – is hydroxyurea. It works in some patient, it’s got some toxicity, but investigators are looking at other drugs that approach treating Sickle Cell in a different manner. This drug, GPT440, I like because one, it’s an oral agent, it’s a pill that patients would need to take once a day, but it’s not a shot, you don’t have to have an IV, you don’t have to take it multiple times a day. And what this drug does is it enhances the affinity of oxygen for hemoglobin, and it doesn’t have as many toxicities as, for example, say hydroxyurea. What’s been shown in England, in adult Sickle Cell patients, for those patients who took this drug over a month, it resulted in a one gram increase in their hemoglobin. That may not sound like much, but when some Sickle Cell patients have hemoglobin routinely as low as five or six, so if you raise it a gram, you’re actually raising their hemoglobin by 20% and then this could help decrease complications. It’s really exciting. We’re actually proudly participating in what we call Part A of the study, and we think that Part B will probably open later this month, February of 2017.

Dr. Smith: Is this multicenter, or is it only at Children’s Mercy?

Dr. Woods: No, it’s multicenter. We were one of two centers initially that got involved. St. Jude’s was the other. I know a lot of times people think St. Jude is a cancer center, but they also treat hematology patients. Now they’ve expanded to about six to ten centers throughout the country, West Coast, Mid-West, St. Jude’s, and a couple on the East Coast.

Dr. Smith: Wow, very exciting work there. Let’s move into another discussion. You are also leading the hospital’s participation in the Sickle Cell Disease Treatment Demonstration Program, and I believe you received a grant from Health Resources and the Services Administration. Tell us about what this program is, what we’re trying to do with it.

Dr. Woods: So this is a regional collaborative, and it involves the States of Missouri, Kansas, Nebraska, and Iowa. As you’ve said, it was funded by Health Resources and Services Administration. It began in 2014. The first three-year cycle will end August of this year. We have three main purposes, but then there may be some things to expand upon that. We’re trying to increase the number of providers that are treating Sickle Cell patients in our region. We’re calling it the Heartland Region, and particularly in rural areas, sometimes that’s difficult. In urban areas, they have centers like ours. We’re trying to increase the number of providers that prescribe or recommend the appropriate use of Hydroxyurea. As I mentioned, it’s the only FDA-Approved modifying therapy that has had success. We’re also trying to better educate providers about Sickle Cell Disease. We’re developing, not only a regional infrastructure for those four states but also a state infrastructure where we share our ideas – what has worked, what hasn’t worked -- we’re trying to just give overall better care through coordination of our service delivery, counseling, like I mentioned, and just using all of the expertise that we’ve already developed and sharing it with others who may not have had the opportunity to have access to these things.

Dr. Smith: Okay, now when you mentioned one of the goals here is to increase the number of providers who can treat Sickle Cell Disease appropriately, so you’re talking about educating the General Practitioner, not necessarily more Hematologists in an area, but taking the Nurse Practitioner, the GP and giving them the education, giving them the skills to treat appropriately. Is that the focus?

Dr. Woods: That is the focus because we believe strongly that all Sickle Cell patients should have Primary Care Providers. There are other problems besides Sickle Cell that they have, but also, instead of having to come two to three hours to our center, or other centers, if they could get their care more locally, and their providers are more comfortable, it would once again improve quality of life, improve the medical care that’s delivered that way. We can try to accomplish this by educational conferences, by Telemedicine, by one-on-one conversations with providers to help develop their expertise and level of comfort.

Dr. Smith: Well, that’s fascinating work, Dr. Woods, and I want to thank you for what you’re doing. Good luck with the GBT440 study. I hope it has the results that you’re looking for and also with the demonstration program. And also, thanks for coming on the show. You’re listening to Transformational Pediatrics with Children’s Mercy Kansas City. For more information, you can go to ChildrensMercy.org, that’s ChildrensMercy.org. I’m Dr. Mike Smith, thanks for listening.